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With the growth of subspecialization in every field of medicine, there are often new therapeutic options for the same disease from different subspecialties. For example, a 3.5 cm hepatocellular carcinoma in the left lobe of the liver in a 72-year-old with Child-Pugh (CP) A cirrhosis and portal hypertension with an Eastern Cooperative Oncology Group performance score of 1 can be treated with a variety of therapeutic options—resection, thermal ablation, bland embolization, chemoembolization, radioembolization, stereotactic body radiation therapy, or some combination of these and medical therapy. The treatment would most likely be governed by local expertise, availability of treatment, affordability, and patient preference. Many would believe that a randomized study would make it easier to decide on therapies. Strict selection criteria in clinical studies, however, limits the generalization of the outcomes in patients outside of the selection criteria. Sorafenib, for example, is approved for treatment of hepatocellular carcinoma as it was proven to improve survival outcomes (by 2.8 months) in CP A patients (with additional specific criteria for platelet count and hepatic and renal function) compared with placebo.[1] It is unknown whether Sorafenib would have similar effect in CP B patients. Systematic reviews, meta-analysis, network meta-analysis, propensity score matching for statistical analysis of observational data are some of the methods applied to assess the comparative effectiveness of various treatments when direct randomized data are not available. It is important to note that the technology and drugs evolve in continuum so frequent analysis of data are required to understand the comparative outcomes of the treatment options.

• References

• 1 Llovet JM, Ricci S, Mazzaferro V. et al; SHARP Investigators Study Group. Sorafenib in advanced hepatocellular carcinoma. N Engl J Med 2008; 359 (04) 378-390 10.1056/NEJMoa0708857
• 2 Yopp AC, Mansour JC, Beg MS. et al. Establishment of a multidisciplinary hepatocellular carcinoma clinic is associated with improved clinical outcome. Ann Surg Oncol 2014; 21 (04) 1287-1295 10.1245/s10434-013-3413-8